Treatment options for chronic immune (idiopathic) thrombocytopenia purpura in children

Abstract

Immune (idiopathic) thrombocytopenic purpura (ITP) in children is usually acute and self-limiting, but may become chronic in 10% to 30% of patients. Salient issues in the treatment of childhood chronic ITP (cITP) include the following: the choice of immunomodulatory agent; the child's desire for unrestricted physical activity; interventions to avoid or defer splenectomy; and, finally, choosing when (and how) to perform splenectomy. Treatment for children with cITP during childhood usually is extrapolated from that for acute ITP. Treatment with pooled intravenous immunoglobulin (IVIg) and anti-D immunoglobulin often gives an acute response followed by a predictable decay of platelet count. Corticosteroids usually lead to a platelet increase; however, the associated adverse effects of chronic usage are generally unsatisfactory for most children and adolescents. With pulsed, high-dose corticosteroids, a durable platelet response is the exception, not the rule. More aggressive immunosuppression is usually reserved for patients who are symptomatic and refractory to the above treatments, including splenectomy. Although the estimated success rate ranges from 70% to 90%, the long-term outcome of splenectomy in children with cITP in not well described. In addition, the risk of fatal postsplenectomy infections is significant. A familiar initial strategy among pediatric hematologists thus involves deferral of splenectomy with the reasonable possibility of spontaneous recovery. Corticosteroids, anti-D, and IVIg are effective, temporizing medical alternatives to splenectomy in treating cITP in children. Quality-of-life measurements in children with cITP may help to stimulate the development of new approaches.